FDA issues draft guidance to streamline approval of personalized gene therapies

The FDA released a draft guidance that outlines an "adapted pathway" for regulating gene‑edited medicines. The new framework is intended to speed the development and approval of personalized gene therapies that target specific genetic mutations.

The concept was first described by former CBER chief Peter Marks in 2024, who warned that a traditional review process could not handle the volume of mutation‑specific products. Current CBER director Karim Mikhail said the guidance moves that vision forward and could simplify the regulatory steps for groups of related therapies.

Industry observers see the guidance as a concrete step toward making individualized genetic medicines more accessible, potentially reducing the time and cost required to bring these treatments to patients.